Clinical translation of TALENS: Treating SCID-X1 by gene editing in iPSCs.
basic_science · Level V
Where this comes from
- Record sourced from PubMed, PMID 25842973.
- Also identified by DOI 10.1016/j.stem.2015.03.009.
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Abstract
Mutations causing X-linked severe combined immunodeficiency (SCID-X1) reduce immune cell populations and function and may be amenable to targeted gene correction strategies. Now in Cell Stem Cell, Menon et al. (2015) correct SCID-X1-related blood differentiation defects by TALEN-mediated genome editing in patient-derived iPSCs, suggesting a possible strategy for autologous cell therapy of SCID-X1.
Medical subject headings
- Genetic Therapy
- Immunotherapy, Adoptive
- Induced Pluripotent Stem Cells
- Killer Cells, Natural
- Precursor Cells, T-Lymphoid
- Regeneration
- Regenerative Medicine
- X-Linked Combined Immunodeficiency Diseases